Mechanisms underlying metabolic and neural defects in zebrafish and human multiple acyl-CoA dehydrogenase deficiency (MADD).

In humans, mutations in electron transfer flavoprotein (ETF) or electron transfer flavoprotein dehydrogenase (ETFDH) lead to MADD/glutaric aciduria type II, an autosomal recessively inherited disorder characterized by a broad spectrum of devastating neurological, systemic and metabolic symptoms. We...

Full description

Saved in:
Bibliographic Details
Main Authors: Yuanquan Song, Mary A Selak, Corey T Watson, Christopher Coutts, Paul C Scherer, Jessica A Panzer, Sarah Gibbs, Marion O Scott, Gregory Willer, Ronald G Gregg, Declan W Ali, Michael J Bennett, Rita J Balice-Gordon
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2009-12-01
Series:PLoS ONE
Online Access:https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0008329&type=printable
Tags: Add Tag
No Tags, Be the first to tag this record!
_version_ 1849695273625845760
author Yuanquan Song
Mary A Selak
Corey T Watson
Christopher Coutts
Paul C Scherer
Jessica A Panzer
Sarah Gibbs
Marion O Scott
Gregory Willer
Ronald G Gregg
Declan W Ali
Michael J Bennett
Rita J Balice-Gordon
author_facet Yuanquan Song
Mary A Selak
Corey T Watson
Christopher Coutts
Paul C Scherer
Jessica A Panzer
Sarah Gibbs
Marion O Scott
Gregory Willer
Ronald G Gregg
Declan W Ali
Michael J Bennett
Rita J Balice-Gordon
author_sort Yuanquan Song
collection DOAJ
description In humans, mutations in electron transfer flavoprotein (ETF) or electron transfer flavoprotein dehydrogenase (ETFDH) lead to MADD/glutaric aciduria type II, an autosomal recessively inherited disorder characterized by a broad spectrum of devastating neurological, systemic and metabolic symptoms. We show that a zebrafish mutant in ETFDH, xavier, and fibroblast cells from MADD patients demonstrate similar mitochondrial and metabolic abnormalities, including reduced oxidative phosphorylation, increased aerobic glycolysis, and upregulation of the PPARG-ERK pathway. This metabolic dysfunction is associated with aberrant neural proliferation in xav, in addition to other neural phenotypes and paralysis. Strikingly, a PPARG antagonist attenuates aberrant neural proliferation and alleviates paralysis in xav, while PPARG agonists increase neural proliferation in wild type embryos. These results show that mitochondrial dysfunction, leading to an increase in aerobic glycolysis, affects neurogenesis through the PPARG-ERK pathway, a potential target for therapeutic intervention.
format Article
id doaj-art-55359c39e9ca49cea285c1631d908dbe
institution DOAJ
issn 1932-6203
language English
publishDate 2009-12-01
publisher Public Library of Science (PLoS)
record_format Article
series PLoS ONE
spelling doaj-art-55359c39e9ca49cea285c1631d908dbe2025-08-20T03:19:50ZengPublic Library of Science (PLoS)PLoS ONE1932-62032009-12-01412e832910.1371/journal.pone.0008329Mechanisms underlying metabolic and neural defects in zebrafish and human multiple acyl-CoA dehydrogenase deficiency (MADD).Yuanquan SongMary A SelakCorey T WatsonChristopher CouttsPaul C SchererJessica A PanzerSarah GibbsMarion O ScottGregory WillerRonald G GreggDeclan W AliMichael J BennettRita J Balice-GordonIn humans, mutations in electron transfer flavoprotein (ETF) or electron transfer flavoprotein dehydrogenase (ETFDH) lead to MADD/glutaric aciduria type II, an autosomal recessively inherited disorder characterized by a broad spectrum of devastating neurological, systemic and metabolic symptoms. We show that a zebrafish mutant in ETFDH, xavier, and fibroblast cells from MADD patients demonstrate similar mitochondrial and metabolic abnormalities, including reduced oxidative phosphorylation, increased aerobic glycolysis, and upregulation of the PPARG-ERK pathway. This metabolic dysfunction is associated with aberrant neural proliferation in xav, in addition to other neural phenotypes and paralysis. Strikingly, a PPARG antagonist attenuates aberrant neural proliferation and alleviates paralysis in xav, while PPARG agonists increase neural proliferation in wild type embryos. These results show that mitochondrial dysfunction, leading to an increase in aerobic glycolysis, affects neurogenesis through the PPARG-ERK pathway, a potential target for therapeutic intervention.https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0008329&type=printable
spellingShingle Yuanquan Song
Mary A Selak
Corey T Watson
Christopher Coutts
Paul C Scherer
Jessica A Panzer
Sarah Gibbs
Marion O Scott
Gregory Willer
Ronald G Gregg
Declan W Ali
Michael J Bennett
Rita J Balice-Gordon
Mechanisms underlying metabolic and neural defects in zebrafish and human multiple acyl-CoA dehydrogenase deficiency (MADD).
PLoS ONE
title Mechanisms underlying metabolic and neural defects in zebrafish and human multiple acyl-CoA dehydrogenase deficiency (MADD).
title_full Mechanisms underlying metabolic and neural defects in zebrafish and human multiple acyl-CoA dehydrogenase deficiency (MADD).
title_fullStr Mechanisms underlying metabolic and neural defects in zebrafish and human multiple acyl-CoA dehydrogenase deficiency (MADD).
title_full_unstemmed Mechanisms underlying metabolic and neural defects in zebrafish and human multiple acyl-CoA dehydrogenase deficiency (MADD).
title_short Mechanisms underlying metabolic and neural defects in zebrafish and human multiple acyl-CoA dehydrogenase deficiency (MADD).
title_sort mechanisms underlying metabolic and neural defects in zebrafish and human multiple acyl coa dehydrogenase deficiency madd
url https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0008329&type=printable
work_keys_str_mv AT yuanquansong mechanismsunderlyingmetabolicandneuraldefectsinzebrafishandhumanmultipleacylcoadehydrogenasedeficiencymadd
AT maryaselak mechanismsunderlyingmetabolicandneuraldefectsinzebrafishandhumanmultipleacylcoadehydrogenasedeficiencymadd
AT coreytwatson mechanismsunderlyingmetabolicandneuraldefectsinzebrafishandhumanmultipleacylcoadehydrogenasedeficiencymadd
AT christophercoutts mechanismsunderlyingmetabolicandneuraldefectsinzebrafishandhumanmultipleacylcoadehydrogenasedeficiencymadd
AT paulcscherer mechanismsunderlyingmetabolicandneuraldefectsinzebrafishandhumanmultipleacylcoadehydrogenasedeficiencymadd
AT jessicaapanzer mechanismsunderlyingmetabolicandneuraldefectsinzebrafishandhumanmultipleacylcoadehydrogenasedeficiencymadd
AT sarahgibbs mechanismsunderlyingmetabolicandneuraldefectsinzebrafishandhumanmultipleacylcoadehydrogenasedeficiencymadd
AT marionoscott mechanismsunderlyingmetabolicandneuraldefectsinzebrafishandhumanmultipleacylcoadehydrogenasedeficiencymadd
AT gregorywiller mechanismsunderlyingmetabolicandneuraldefectsinzebrafishandhumanmultipleacylcoadehydrogenasedeficiencymadd
AT ronaldggregg mechanismsunderlyingmetabolicandneuraldefectsinzebrafishandhumanmultipleacylcoadehydrogenasedeficiencymadd
AT declanwali mechanismsunderlyingmetabolicandneuraldefectsinzebrafishandhumanmultipleacylcoadehydrogenasedeficiencymadd
AT michaeljbennett mechanismsunderlyingmetabolicandneuraldefectsinzebrafishandhumanmultipleacylcoadehydrogenasedeficiencymadd
AT ritajbalicegordon mechanismsunderlyingmetabolicandneuraldefectsinzebrafishandhumanmultipleacylcoadehydrogenasedeficiencymadd